On September 13, 2018 Boehringer Ingelheim reported that it has acquired all shares of ViraTherapeutics, a biopharmaceutical company specializing in the development of oncolytic viral therapies (Press release, Boehringer Ingelheim, SEP 13, 2018, View Source [SID1234529439]). ViraTherapeutics developed the lead candidate VSV-GP (Vesicular Stomatitis Virus (VSV) with modified glycoprotein (GP)), which is being investigated alone and in combination with other therapies. The total transaction value of EUR 210 million is based on an option and share purchase agreement signed between the companies in August 2016.

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Oncolytic viral therapy is a cancer treatment approach with two modes of action. First, the virus specifically replicates in and kills cancer cells. Second, viral infection stimulates the immune system to recognize these same cancer cells, leading to immune-mediated killing of both infected and non-infected cancer cells, further enhancing tumor control. Boehringer Ingelheim and ViraTherapeutics are working to develop a next generation oncolytic viral therapy platform. The lead investigational candidate leveraging the platform, VSV-GP, has shown promising results in pre-clinical models, especially in combination with key immune modulatory principles Boehringer Ingelheim is developing.

"The acquisition of ViraTherapeutics with its exciting oncolytic virus platform is the conclusion of a trusting and close cooperation over two years," said Dr. Heinz Schwer, CEO of ViraTherapeutics. "We are highly optimistic that our VSV-based development programs and technology will complement Boehringer Ingelheim’s immuno-oncology franchise and will serve as a source of innovative, new treatment options for patients living with cancer."

"I want to thank the team around scientific founder Dorothee von Laer, CEO Heinz Schwer and COO Lisa Egerer for their dedication to research and scientific progress that led to a productive collaboration with Boehringer Ingelheim and ultimately to the early exercise of the purchase option," said Dr. Klaus Schollmeier, Chairman of ViraTherapeutics’ advisory board. "I also want to thank the investors and my fellow board members for their hands-on commitment to this project. I am convinced that ViraTherapeutics’ research will become core to Boehringer Ingelheim’s oncology product pipeline."

Using a dual approach for potential treatment options, specifically combining immuno-oncology approaches with tumor cell-directed treatments, is central to Boehringer Ingelheim’s cancer immunology research strategy. Oncolytic virus-based therapies are consistent with and complement that strategy.

"Our approach is rooted in transforming ‘cold’ tumors – or immunologically inactive tumors that are not responsive to the checkpoint blockers – to ‘hot‘ tumors – those that are most susceptible to immune system attack," said Dr. Michel Pairet, member of Boehringer Ingelheim’s Board of Managing Directors responsible for Boehringer Ingelheim’s Research and Development. "We are committed to investing in early research with promise and where our expertise best complements the strengths of our partners. Together, we aim to discover breakthrough medical treatments to transform the lives of patients and win the fight against cancer."

ViraTherapeutics was a portfolio company of the two venture investors EMBL Ventures and the Boehringer Ingelheim Venture Fund (BIVF). BIVF is focused on strategic investment in highly innovative biotechnology and start-up companies to help drive innovation in medical science. The BIVF has EUR 250 million under management and currently supervises a portfolio of 22 active companies and is one of the most active investors in immuno-oncology world-wide.

On September 13 Array BioPharma Inc. (NASDAQ: ARRY) reported the publication of detailed overall survival (OS) results from the COLUMBUS trial in The Lancet Oncology (Press release, Array BioPharma, SEP 13, 2018, View Source [SID1234529437]). The pivotal Phase 3 trial evaluated the efficacy and safety of the combination of BRAFTOVI+ MEKTOVI compared to vemurafenib monotherapy in patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation. BRAFTOVI + MEKTOVI reduced the risk of death compared to treatment with vemurafenib [hazard ratio (HR) of 0.61, (95% CI 0.47-0.79, p <0.0001)] in the planned analysis of OS. Median OS was 33.6 months for patients treated with the combination, compared to 16.9 months for patients treated with vemurafenib as a monotherapy. The analysis of OS and updated efficacy and safety data extend findings of previously announced results.

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Based on data from the previously reported primary analysis of the COLUMBUS trial, the U.S. Food and Drug Administration (FDA) approved BRAFTOVI capsules in combination with MEKTOVI tablets on June 27, 2018 for the treatment of patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for the treatment of patients with wild-type BRAF melanoma. Further, on July 16, 2018, Array submitted supplementary New Drug Applications to seek inclusion of OS data from the COLUMBUS trial in the BRAFTOVI and MEKTOVI labels.

Mature median progression-free survival (mPFS) for BRAFTOVI + MEKTOVI was 14.9 months (95% CI 11.0–20.2) compared to vemurafenib with 7.3 months (5.6–7.9), [HR 0.51, (95% CI 0.39–0.67; two-sided p<0.0001)].

"The publication of updated findings from the COLUMBUS trial in The Lancet Oncology underscores the value that BRAFTOVI + MEKTOVI bring to patients with BRAF-mutant melanoma," said Keith T. Flaherty, M.D., Director of the Termeer Center for Targeted Therapy, Massachusetts General Hospital Cancer Center and Professor of Medicine, Harvard Medical School. "Based on the observed efficacy and tolerability, this targeted combination is an important new treatment option for patients with this life-threatening form of melanoma."

Use of post-trial immunotherapy was limited and consistent with other published pivotal trials of BRAF + MEK inhibitors in advanced BRAF-mutant melanoma. The manuscript also noted that the performance of vemurafenib, the control arm, was consistent across efficacy endpoints with its performance in other trials of BRAF + MEK inhibitor combinations where it also served as a control. Finally, the authors conclude the data represent a new benchmark for BRAF + MEK inhibitors in metastatic or unresectable BRAF-mutant melanoma.

With 18 months of additional follow-up relative to the initial publication of COLUMBUS results, the safety profile of the combination remained generally consistent with the prior report. Observed grade 3 or 4 adverse events in more than 5% of patients with BRAFTOVI + MEKTOVI were increased gamma-glutamyltransferase (9%), increased blood creatine phosphokinase (7%) and hypertension (6%). Additional safety information can be found in the manuscript and in the Important Safety Information below.

About BRAF-mutant Metastatic Melanoma
Melanoma develops when unrepaired DNA damage to skin cells triggers mutations that may lead them to multiply and form malignant tumors. Metastatic melanoma is the most serious and life-threatening type of skin cancer and is associated with low survival rates. [1, 2] There are a variety of gene mutations that can lead to metastatic melanoma. The most common genetic mutation in metastatic melanoma is BRAF. There are about 200,000 new cases of melanoma diagnosed worldwide each year, approximately half of which have BRAF mutations, a key target in the treatment of metastatic melanoma. [1, 3, 4, 5]

About BRAFTOVI + MEKTOVI
BRAFTOVI is an oral small molecule BRAF kinase inhibitor and MEKTOVI is an oral small molecule MEK inhibitor which target key enzymes in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Inappropriate activation of proteins in this pathway has been shown to occur in many cancers including melanoma, colorectal cancer, non-small cell lung cancer and others. In the U.S., BRAFTOVI + MEKTOVI are approved for the treatment of unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for treatment of patients with wild-type BRAF melanoma.

Array has exclusive rights to BRAFTOVI and MEKTOVI in the U.S. and Canada. Array has granted Ono Pharmaceutical Co, Ltd. exclusive rights to commercialize both products in Japan and South Korea, Medison exclusive rights to commercialize both products in Israel and Pierre Fabre exclusive rights to commercialize both products in all other countries, including Europe, Asia (excluding Japan and South Korea) and Latin America.

BRAFTOVI and MEKTOVI are not approved outside of the U.S. The European Medicines Agency (EMA), the Swiss Medicines Agency (Swissmedic) and the Australian Therapeutic Goods Administration (TGA), are currently reviewing the Marketing Authorization Applications submitted by Pierre Fabre, and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) is currently reviewing the Manufacturing and Marketing Approval Applications for submitted by Ono Pharmaceutical Co, Ltd.

About COLUMBUS
The COLUMBUS trial (NCT01909453) is a two-part, international, randomized, open label Phase 3 trial evaluating the efficacy and safety of BRAFTOVI (encorafenib) in combination with MEKTOVI (binimetinib) compared to vemurafenib and encorafenib monotherapy in 921 patients with locally advanced, unresectable or metastatic melanoma with BRAFV600 mutation. The primary endpoint of the trial was mPFS; all secondary efficacy analyses, including the prospectively planned analysis of overall survival, are descriptive in nature. Over 200 sites across North America, Europe, South America, Asia and Australia participated in the trial.

BRAFTOVI + MEKTOVI Indications and Usage
BRAFTOVI (encorafenib) and MEKTOVI (binimetinib) are kinase inhibitors indicated for use in combination for the treatment of patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by an FDA-approved test.

Limitations of Use: BRAFTOVI is not indicated for the treatment of patients with wild-type BRAF melanoma.

BRAFTOVI + MEKTOVI Important Safety Information
The information below applies to the safety of the combination of BRAFTOVI and MEKTOVI unless otherwise noted.

Warnings and Precautions
New Primary Malignancies: New primary malignancies, cutaneous and non-cutaneous malignancies can occur. In the COLUMBUS trial, cutaneous squamous cell carcinoma, including keratoacanthoma, occurred in 2.6% and basal cell carcinoma occurred in 1.6% of patients. Perform dermatologic evaluations prior to initiating treatment, every 2 months during treatment, and for up to 6 months following discontinuation of treatment. Discontinue BRAFTOVI for RAS mutation-positive non-cutaneous malignancies.

Tumor Promotion in BRAF Wild-Type Tumors: Confirm evidence of BRAFV600E or BRAFV600K mutation prior to initiating BRAFTOVI.

Cardiomyopathy: In the COLUMBUS trial, cardiomyopathy occurred in 7% and Grade 3 left ventricular dysfunction occurred in 1.6% of patients. Cardiomyopathy resolved in 87% of patients. Assess left ventricular ejection fraction by echocardiogram or MUGA scan prior to initiating treatment, 1 month after initiating treatment, and then every 2 to 3 months during treatment. The safety has not been established in patients with a baseline ejection fraction that is either below 50% or below the institutional lower limit of normal.

Venous Thromboembolism (VTE): In the COLUMBUS trial, VTE occurred in 6% of patients, including 3.1% of patients who developed pulmonary embolism.

Hemorrhage: In the COLUMBUS trial, hemorrhage occurred in 19% of patients and ≥Grade 3 hemorrhage occurred in 3.2% of patients. Fatal intracranial hemorrhage in the setting of new or progressive brain metastases occurred in 1.6% of patients.

Ocular Toxicities: In the COLUMBUS trial, serous retinopathy occurred in 20% of patients; 8% were retinal detachment and 6% were macular edema. Symptomatic serous retinopathy occurred in 8% of patients with no cases of blindness. In patients with BRAF mutation-positive melanoma across multiple clinical trials, 0.1% of patients experienced retinal vein occlusion (RVO). Permanently discontinue MEKTOVI in patients with documented RVO. In COLUMBUS, uveitis, including iritis and iridocyclitis, was reported in 4% of patients. Assess for visual symptoms at each visit. Perform ophthalmic evaluation at regular intervals and for any visual disturbances.

Interstitial Lung Disease (ILD): ILD, including pneumonitis, occurred in 0.3% of patients with BRAF mutation-positive melanoma across multiple clinical trials. Assess new or progressive unexplained pulmonary symptoms or findings for possible ILD.

Hepatotoxicity: In the COLUMBUS trial, the incidence of Grade 3 or 4 increases in liver function laboratory tests was 6% for alanine aminotransferase (ALT) and 2.6% for aspartate aminotransferase (AST). Monitor liver laboratory tests before and during treatment and as clinically indicated.

Rhabdomyolysis: In the COLUMBUS trial, elevation of laboratory values of serum creatine phosphokinase (CPK) occurred in 58% of patients. Rhabdomyolysis was reported in 0.1% of patients with BRAF mutation-positive melanoma across multiple clinical trials. Monitor CPK periodically and as clinically indicated.

QTc Prolongation: In the COLUMBUS trial, an increase in QTcF to >500 ms was measured in 0.5% (1/192) of patients. Monitor patients who already have or who are at significant risk of developing QTc prolongation. Correct hypokalemia and hypomagnesemia prior to and during BRAFTOVI administration. Withhold, reduce dose, or permanently discontinue for QTc >500 ms.

Embryo-Fetal Toxicity: BRAFTOVI or MEKTOVI can cause fetal harm when administered to pregnant women. Nonhormonal contraceptives should be used during treatment and for at least 30 days after the final dose for patients taking BRAFTOVI + MEKTOVI.

Adverse Reactions
The most common adverse reactions (≥20%, all Grades, in the COLUMBUS trial) were: fatigue, nausea, diarrhea, vomiting, abdominal pain, arthralgia, myopathy, hyperkeratosis, rash, headache, constipation, visual impairment, serous retinopathy.

In the COLUMBUS Trial, the most common laboratory abnormalities (≥20%, all Grades) included: increased creatinine, increased CPK, increased gamma glutamyl transferase, anemia, increased ALT, hyperglycemia, increased AST, and increased alkaline phosphatase.

Drug interactions
Avoid concomitant use of strong or moderate CYP3A4 inhibitors or inducers and sensitive CYP3A4 substrates with BRAFTOVI. Modify BRAFTOVI dose if concomitant use of strong or moderate CYP3A4 inhibitors cannot be avoided.

Please see full Prescribing Information for BRAFTOVI and full Prescribing Information for MEKTOVI for additional information [6,7]. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Array at 1-844-Rx-Array (1-844-792-7729).

On September 13, 2018 SELLAS Life Sciences Group, Inc. (Nasdaq:SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has approved orphan medicinal product designation (OMPD) for galinpepimut-S (GPS), the Company’s lead product candidate, for the treatment of multiple myeloma (MM) (Press release, Sellas Life Sciences, SEP 13, 2018, View Source [SID1234529423]). GPS is licensed from Memorial Sloan Kettering Cancer Center and targets the Wilms Tumor 1 (WT1) protein, which is present in an array of tumor types. GPS has also been granted orphan drug designation and fast track designation by the U.S. Food and Drug Administration (FDA) for the treatment of MM.

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"This OMPD endorsement by the COMP of the EMA for GPS in MM complements the orphan designation awarded by the US FDA for this product in the same indication," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The results from our open-label Phase 2 study reinforce the potential of GPS to serve as a therapy for high-risk MM patients in the post-autotransplant maintenance setting. The innovative nature and unique mechanism of action for GPS provide a promising potential addition to the current arsenal of therapies in this indication. We continue to work closely with the FDA and EMA, as well as multiple myeloma KOLs to further advance the clinical development of GPS in this malignancy and look forward to gaining further insights on the potential therapeutic role of GPS in high-risk MM patients."

The EMA orphan medicinal product designation is granted to medicines being developed for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition with a prevalence of not more than five in 10,000 people in the European Union. Orphan designations are granted by decisions of the European Commission based on opinions from the Committee for Orphan Medicinal Products within EMA. EMA orphan drug designation benefits include protocol assistance, access to the EU centralized authorization procedure, reduced EU regulatory filing fees and 10 years of market exclusivity across the EU.

About the Phase 2 Trial of GPS in Multiple Myeloma

The open-label Phase 2 study consisted of 19 patients with multiple myeloma who had high-risk cytogenetics at initial diagnosis and remained at least minimal residual disease (MRD)-positive after a successful autologous stem cell transplant ("ASCT"). GPS was administered to patients in the study who achieved a stable disease or better status (per International Myeloma Working Group criteria) following ASCT. GPS was evaluated as consolidation therapy (on top of lenalidomide or bortezomib) to potentially stimulate a highly-specific immune response against WT1 in order to prevent or delay myeloma progression. Median progression-free survival (PFS) of 23.6 months was reported in this high-risk disease setting, compared to historically inferior outcomes while on an immunomodulatory drug (IMID) or proteasome inhibitor post-ASCT maintenance. Median overall survival has not been reached to date. GPS stimulated time-dependent and robust CD4+ T cell or CD8+ T cell immune responses (IRs) specific for all four WT1 peptides within GPS, two of which are heteroclitic (mutated, by design). In addition, GPS stimulated similar IRs against the two counterpart native peptides. The IRs were confirmed in up to 91% of patients across HLA allele types, with multivalent IRs emerging in up to 64% of patients. Multifunctional cross-epitope T cell reactivity was observed in 75% of patients to antigenic epitopes against which hosts were not specifically immunized, in a pattern akin to epitope spreading. A link of clinical activity to antigen-specific immune responses was suggested.

About Galinpepimut-S (GPS)

GPS is a heteroclitic multivalent, multi-peptide cancer immunotherapeutic agent composed of four peptides, addressing over 20 epitopes, and derived from the WT1 protein, which has been ranked by the National Cancer Institute as a top priority among cancer antigens for immunotherapy. Importantly, because the WT1 antigen is overexpressed in many malignancies, and is not found in most normal tissues, GPS has the potential to be a broad immunotherapy, effective across a multitude of diverse cancer types and patient populations.

On September 13, 2018 AngioDynamics, Inc. (NASDAQ: ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, peripheral vascular disease, and oncology, reported that it will report financial results for the first quarter of fiscal year 2019 before the market open on Thursday, September 27, 2018 (Press release, AngioDynamics, SEPT 13, 2018, View Source [SID1234529419]). The Company’s management will host a conference call at 8:00 a.m. ET the same day to discuss the results.

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To participate in the conference call, dial 1-877-407-0784 (domestic) or 1-201-689-8560 (international) and refer to the passcode 13683219.

This conference call will also be webcast and can be accessed from the "Investors" section of the AngioDynamics website at www.angiodynamics.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A recording of the call will also be available from 11:00 a.m. ET on Thursday, September 27, 2018, until 11:59 p.m. ET on Thursday, October 4, 2018. To hear this recording, dial 1-844-512-2921 (domestic) or 1-412-317-6671 (international) and enter the passcode 13683219.

On September 13, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that updated clinical data from the Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study evaluating selinexor, the Company’s lead, oral Selective Inhibitor of Nuclear Export (SINE) compound, in heavily pretreated patients with penta-refractory multiple myeloma, were presented during an oral session at the Society of Hematologic Oncology (SOHO) 2018 Annual Meeting on September 13, 2018, in Houston (Press release, Karyopharm, SEPT 13, 2018, View Source [SID1234529418]). Sundar Jagannath, MD, Director of the Multiple Myeloma Program, Professor of Medicine (Hematology and Medical Oncology) at Tisch Cancer Institute at Mount Sinai School of Medicine, and principal investigator of the STORM study, presented the data in a session entitled, "Phase 2b Results of the STORM Study: Oral Selinexor plus Low Dose Dexamethasone (Sd) in Patients with Penta-Refractory Myeloma."

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"The additional Phase 2b clinical results presented today are very encouraging for the patients suffering from penta-refractory multiple myeloma and their families. Most notably, the overall response rate (ORR) for patients treated with oral selinexor and dexamethasone (dex; Sd) was 26.2% with median duration of response (DOR) of 4.4 months based on the Independent Review Committee (IRC) assessment, along with a median overall survival (OS) across the entire study of 8.6 months," said Dr. Jagannath. "Of particular significance, for the nearly 40% of patients who had a minimal response (MR) or better, the median survival was 15.6 months, which provided the opportunity for a meaningful clinical benefit for patients on the STORM study with advanced penta-refractory myeloma that is difficult to treat."

Dan Vogl, MD, MSCE, Assistant Professor of Medicine at the Hospital of the University of Pennsylvania, commented, "The results from the Phase 2b STORM study showed that selinexor resulted in a meaningful clinical benefit in this heavily pretreated patient population. This includes patients treated with the most modern combination therapies and most exciting experimental therapies. For example, the overall response rate was 29.1% in patients who had previously been treated with daratumumab combination regimens, and the two patients on the STORM study who had previously received investigational CAR-T cell therapy both achieved partial responses on selinexor and dexamethasone. These results provide further evidence that selective inhibition of nuclear export could be an effective strategy for myeloma therapy and of selinexor’s potential to be a new option for patients with penta-refractory multiple myeloma."

"Patients with highly resistant myeloma have very few treatment options available, which underscores the urgent need for the advancement of therapies with novel mechanisms, like selinexor," said Sharon Shacham, PhD, Founder, President and Chief Scientific Officer of Karyopharm. "The 26.2% ORR from the STORM study is particularly meaningful considering that 96% of the patients had myeloma refractory to Kyprolis, Pomalyst and Darzalex, and nearly 70% of patients had disease that was confirmed to be refractory to all five of the standard of care myeloma drugs, Revlimid, Velcade, Pomalyst, Kyprolis, and Darzalex. These results reinforce the potential of selinexor in this difficult to treat patient population. Following our recent submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for selinexor with low dose dexamethasone, we are making great strides in building our key commercial capabilities as we prepare for a potential initial market launch, which could be as early as the first half of 2019. We also remain on track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the first quarter of 2019 for conditional approval in the same disease indication."

Karyopharm has submitted an NDA to the FDA, with a request for accelerated approval for oral selinexor with low dose dexamethasone as a new treatment for patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. The Company also plans to submit a MAA to the EMA in the first quarter of 2019 with a request for conditional approval. In parallel, Karyopharm is conducting the pivotal, randomized Phase 3 BOSTON study evaluating selinexor in combination with the proteasome inhibitor Velcade and dex (SVd) for the treatment of patients with multiple myeloma who have had one to three prior lines of therapy. The Company expects to complete enrollment in the BOSTON study by the end of 2018, with top-line data anticipated in 2019. Assuming a positive outcome, Karyopharm plans to use the results from the BOSTON study to support an application for full approval of selinexor in relapsed/refractory multiple myeloma. Development of selinexor in other disease indications, including diffuse large B-cell lymphoma, liposarcoma, endometrial cancer and other malignancies remains on track.

Phase 2b STORM Results

These clinical results are from Part 2 of the international, multi-center, single-arm Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study, which enrolled 122 heavily pretreated patients (median of seven prior treatment regiments) with penta-refractory myeloma. Each patient started 80mg oral selinexor twice weekly in combination with low-dose dexamethasone (dex; 20mg twice weekly). Patients with penta-refractory myeloma have previously received the two proteasome inhibitors (PIs), Velcade (bortezomib) and Kyprolis (carfilzomib), the two immunomodulatory drugs (IMiDs), Revlimid (lenalidomide) and Pomalyst (pomalidomide), and the anti-CD38 monoclonal antibody Darzalex (daratumumab), as well as alkylating agents, and their disease is refractory to glucocorticoids, at least one PI, at least one IMiD, Darzalex and their most recent therapy.

For the STORM study’s primary objective, oral selinexor achieved a 26.2% ORR, which included two stringent complete responses (sCRs), six very good partial responses (VGPRs) and 24 partial responses (PRs) in these patients with penta-refractory myeloma. The two sCRs were negative for minimal residual disease, one at the level of 1×10-6 and one at 1×10-4; this is particularly significant in this penta-refractory population. The ORR in patients who had previously received Darzalex combination therapy (n=86) was 29.1%. The Disease Control Rate for patients who had achieved stable disease or better was 78.6%. All responses were confirmed by an IRC. Median progression-free survival (PFS) was 3.7 months and the median DOR was 4.4 months (range <1 to 9.9 months). Median OS across the study was 8.6 months. Median OS in the ~40% of patients with at least a MR on selinexor + dex was 15.6 months compared to a median OS of 1.7 months in patients whose disease progressed or were not evaluable (p<0.0001). The short median OS of patients with no response to selinexor is consistent with the lack of available effective therapies for the very heavily pretreated population who entered the study.

Across the relevant patient population, side effects of oral selinexor were generally predictable and often managed with dose adjustments and/or supportive care, with safety results that were consistent with those previously reported from Part 1 of this study (Vogl et al., J Clin Oncol, 2018) and from other selinexor studies. As anticipated, the most common non-hematologic treatment-related adverse events (AEs) were largely Grade 1/2 and included fatigue (70%), nausea (69%), anorexia (52%) and weight loss (47%). The most common Grade 3/4 AEs were cytopenias (thrombocytopenia (54%) and anemia (29%)) and were generally not associated with clinical sequelae. No significant major organ toxicities were observed, and bleeding and infection rates were low.

Conference Call Information

Karyopharm will host a conference call tomorrow, Friday, September 14, 2018, at 8:00 a.m. Eastern Time, to discuss the Phase 2b STORM clinical data presented at the SOHO 2018 Annual Meeting. The call will feature recognized myeloma experts Drs. Sundar Jagannath and Dan Vogl, along with members of the Karyopharm executive leadership team. To access the conference call, please dial (855) 437-4406 or (484) 756-4292 (international) at least five minutes prior to the start time and refer to conference ID: 8474737. The call will also be webcast live on the Company’s website, View Source An audio recording of the call will be available under "Events & Presentations" in the "Investors" section of Karyopharm’s website approximately two hours after the event.